Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome (PWSGUT)
Primary Purpose
Prader-Willi Syndrome, Obesity
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NBT-NM108
Sponsored by
About this trial
This is an interventional treatment trial for Prader-Willi Syndrome
Eligibility Criteria
Inclusion Criteria:
- Aged between 18-35 (inclusive)
- Confirmed PWS with genetic testing
- No growth hormone treatment in the previous 6 months
- Body weight < 300 lbs.
Exclusion Criteria:
- History of other gastrointestinal disorders such as small intestinal bacterial overgrowth, celiac disease, inflammatory bowel disease, or irritable bowel syndrome.
- Pregnancy or breastfeeding
- Prior gastrointestinal or bariatric surgery
- Immunocompromised e.g., cancer treatment, bone marrow/organ transplant, immune deficiency, poorly controlled HIV/AIDS, prolonged use of steroids or other immunosuppressant medications
- Antibiotic administration in the previous 30 days
- Participation in other weight-loss programs in the previous 3 months.
- Administration of pre/probiotic supplements or antibiotics.
- Growth hormone administration in the previous 6 months
- Must have access to a smartphone, tablet, computer, or other qualifying internet-enabled device and be able to follow instructions.
- Individuals who are not proficient in English
- Contraindications for MRI scanning, including Ferrous material implanted in or on the body, including flakes or filings, surgical clips, bullets, or electrical devices such as a pacemaker, or nonremovable ferrous jewelry (fillings in teeth and permanent retainers are permitted). Individuals with surgical pins or plates above the neck are excluded. Surgical pins or plates below the neck are exclusions, except when the material is fixed to bone, and considered acceptable by the Reference Manual for Magnetic Resonance Safety. Implants and Devices, 2020 Edition. Almost all recent orthopedic implants are made of materials that are not ferromagnetic and therefore are safe for scanning, and even though some screws are still made of ferromagnetic materials these are firmly screwed into bone. In cases where the material is unknown or deemed unsafe for scanning by the Reference Manual for Magnetic Resonance Safety. Implants and Devices, the participant will be excluded. History of eye injury involving metallic materials, shavings in eyes, or welding without a face mask. Lead/iron tattoos and tattoos performed by a nonprofessional artist if the pigment material is unknown. Claustrophobia (history of significant anxiety in closed places).
- Back problem that would prevent the subject from laying still comfortably for up to 60 minutes.
Sites / Locations
- Robert Wood Johnson University Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Experimental Arm
Arm Description
All participants will receive NBT-NM108 prepared as muffin (each contains 30 g of the product) for 4 weeks. The dosage will be 2 muffins a day. This dosage of NBT-NM108 will provide 24 g/day of dietary fibers.
Outcomes
Primary Outcome Measures
Gut microbiota composition
Network analysis will be employed to identify guild-level structure in the gut microbiota. When supplemented with dietary fiber, gut bacteria exhibited co-abundance patterns and formed genome interaction groups (GIGs) or "guilds" (an ecological group in which members exploit an environmental resource in a similar way and show co-abundant behavior).
Gut microbiota composition
Network analysis will be employed to identify guild-level structure in the gut microbiota. When supplemented with dietary fiber, gut bacteria exhibited co-abundance patterns and formed genome interaction groups (GIGs) or "guilds" (an ecological group in which members exploit an environmental resource in a similar way and show co-abundant behavior).
Secondary Outcome Measures
Weight
Weight in lbs
Weight
Weight in lbs
Weight
Weight in lbs
Weight
Weight in lbs
Weight
Weight in lbs
Ghrelin level
fasting and post-prandial hormone level measured in mg/dl
Ghrelin level
fasting and post-prandial hormone level measured in mg/dl
Insulin level
fasting and post-prandial hormone level measured in mg/dl
Insulin level
fasting and post-prandial hormone level measured in mg/dl
Glucose level
fasting and post-prandial hormone level measured in mg/dl
Glucose level
fasting and post-prandial hormone level measured in mg/dl
Functional MRI
functional MRI in the resting state and post-prandial state to assess functional connectivity of feeding related networks, with a specific focus on connectivity between the hypothalamus, insula, anterior cingulate, ventromedial/orbitofrontal prefrontal cortex and amygdala. The output is an image of brain activation.
Functional MRI
functional MRI in the resting state and post-prandial state to assess functional connectivity of feeding related networks, with a specific focus on connectivity between the hypothalamus, insula, anterior cingulate, ventromedial/orbitofrontal prefrontal cortex and amygdala. The output is an image of brain activation.
24 hour dietary recall
24 hour dietary recall recorded in MyFitnessPal application measured in kcal. It is anticipated that the calorie count decreases from week 0 to week 4.
24 hour dietary recall
24 hour dietary recall recorded in MyFitnessPal application measured in kcal. It is anticipated that the calorie count decreases from week 0 to week 4.
Hyperphagia questionnaire
Appetite behavior measured by questionnaire measured by likert scale. The scores should improve from week 0 to week 4. Higher scores indicate worse hyperphagia. Lowest score is 13 and highest score is 65.
Hyperphagia questionnaire
Appetite behavior measured by questionnaire measured by likert scale. The scores should improve from week 0 to week 4. Higher scores indicate worse hyperphagia. Lowest score is 13 and highest score is 65.
Full Information
NCT ID
NCT05541003
First Posted
September 1, 2022
Last Updated
August 18, 2023
Sponsor
Rutgers, The State University of New Jersey
1. Study Identification
Unique Protocol Identification Number
NCT05541003
Brief Title
Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome
Acronym
PWSGUT
Official Title
Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
January 6, 2023 (Actual)
Primary Completion Date
May 29, 2023 (Actual)
Study Completion Date
May 30, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Rutgers, The State University of New Jersey
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
5. Study Description
Brief Summary
This study aims to use a high-fiber supplementation, an intervention known to create shifts in the gut microbiota towards a healthier structure, to explore the relationship between gut microbiota, appetite control and feeding behavior in PWS patients.
Detailed Description
This study is recruiting PWS patients aged 18-35 years who have not received growth hormone treatment in the previous 6 months. Study candidates will be recruited from Robert Wood Johnson. Participants will attend a baseline visit at the Center for Advanced Human Brain Imaging Research (CAHBIR) at Rutgers University, during which functional magnetic resonance imaging (fMRI) coupled with a meal test will be performed to assess peripheral and central feeding pathways (7, 8). fMRI scans will be performed during resting state to assess functional connectivity of feeding related networks, with a specific focus on connectivity between the hypothalamus, insula, anterior cingulate, ventromedial/orbitofrontal prefrontal cortex and amygdala (9, 10). Activation to food (vs non-food) images will be assessed to index responsivity to appetitive feeding networks including the above regions and the ventral striatum using paradigms that have previously been found to be sensitive to trait and state in multiple studies (11-13). Thereafter participants will consume a liquid meal (525 kcal; Ensure Plus, Abbott). fMRI will be repeated immediately after the meal. Participants will obtain laboratory work coupled with a meal test to assess satiety hormones and inflammatory markers. Fasting blood draw will be taken, and the participants will consume a liquid meal (525 kcal; Ensure Plus, Abbot) followed by blood draws at 30, 60, 120, 180 and 240 min post-meal. Upon completion of baseline testing and providing a fecal sample, participants will consume NBT-NM108 (a mixture of inulin, Fibersol-2, and brans of oat, wheat, corn and sorghum; Notitia Biotechnologies) daily for 4 weeks. At the completion of the 4-week intervention, all sampling and testing will be repeated as per baseline. At baseline and end of intervention, participants will be interviewed with appetite and physical activity questionnaires (Appendix C and G) and requested to complete a 24-h food recall using MyFitnessPal, a mobile health app on their phone.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome, Obesity
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Experimental Arm
Arm Type
Experimental
Arm Description
All participants will receive NBT-NM108 prepared as muffin (each contains 30 g of the product) for 4 weeks. The dosage will be 2 muffins a day. This dosage of NBT-NM108 will provide 24 g/day of dietary fibers.
Intervention Type
Drug
Intervention Name(s)
NBT-NM108
Intervention Description
All patients will consume NBT-NM108 in the form of 2 muffins daily.
Primary Outcome Measure Information:
Title
Gut microbiota composition
Description
Network analysis will be employed to identify guild-level structure in the gut microbiota. When supplemented with dietary fiber, gut bacteria exhibited co-abundance patterns and formed genome interaction groups (GIGs) or "guilds" (an ecological group in which members exploit an environmental resource in a similar way and show co-abundant behavior).
Time Frame
Weeks 0
Title
Gut microbiota composition
Description
Network analysis will be employed to identify guild-level structure in the gut microbiota. When supplemented with dietary fiber, gut bacteria exhibited co-abundance patterns and formed genome interaction groups (GIGs) or "guilds" (an ecological group in which members exploit an environmental resource in a similar way and show co-abundant behavior).
Time Frame
Week 4
Secondary Outcome Measure Information:
Title
Weight
Description
Weight in lbs
Time Frame
Week 0
Title
Weight
Description
Weight in lbs
Time Frame
Week 1
Title
Weight
Description
Weight in lbs
Time Frame
Week 2
Title
Weight
Description
Weight in lbs
Time Frame
Week 3
Title
Weight
Description
Weight in lbs
Time Frame
Week 4
Title
Ghrelin level
Description
fasting and post-prandial hormone level measured in mg/dl
Time Frame
Week 0 and week 4
Title
Ghrelin level
Description
fasting and post-prandial hormone level measured in mg/dl
Time Frame
Week 4
Title
Insulin level
Description
fasting and post-prandial hormone level measured in mg/dl
Time Frame
Week 0 and week 4
Title
Insulin level
Description
fasting and post-prandial hormone level measured in mg/dl
Time Frame
Week 4
Title
Glucose level
Description
fasting and post-prandial hormone level measured in mg/dl
Time Frame
Week 0 and week 4
Title
Glucose level
Description
fasting and post-prandial hormone level measured in mg/dl
Time Frame
Week 4
Title
Functional MRI
Description
functional MRI in the resting state and post-prandial state to assess functional connectivity of feeding related networks, with a specific focus on connectivity between the hypothalamus, insula, anterior cingulate, ventromedial/orbitofrontal prefrontal cortex and amygdala. The output is an image of brain activation.
Time Frame
Week 0
Title
Functional MRI
Description
functional MRI in the resting state and post-prandial state to assess functional connectivity of feeding related networks, with a specific focus on connectivity between the hypothalamus, insula, anterior cingulate, ventromedial/orbitofrontal prefrontal cortex and amygdala. The output is an image of brain activation.
Time Frame
Week 4
Title
24 hour dietary recall
Description
24 hour dietary recall recorded in MyFitnessPal application measured in kcal. It is anticipated that the calorie count decreases from week 0 to week 4.
Time Frame
Week 0 and week 4
Title
24 hour dietary recall
Description
24 hour dietary recall recorded in MyFitnessPal application measured in kcal. It is anticipated that the calorie count decreases from week 0 to week 4.
Time Frame
Week 4
Title
Hyperphagia questionnaire
Description
Appetite behavior measured by questionnaire measured by likert scale. The scores should improve from week 0 to week 4. Higher scores indicate worse hyperphagia. Lowest score is 13 and highest score is 65.
Time Frame
Week 0 and week 4
Title
Hyperphagia questionnaire
Description
Appetite behavior measured by questionnaire measured by likert scale. The scores should improve from week 0 to week 4. Higher scores indicate worse hyperphagia. Lowest score is 13 and highest score is 65.
Time Frame
Week 4
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
Aged between 18-35 (inclusive)
Confirmed PWS with genetic testing
No growth hormone treatment in the previous 6 months
Body weight < 300 lbs.
Exclusion Criteria:
History of other gastrointestinal disorders such as small intestinal bacterial overgrowth, celiac disease, inflammatory bowel disease, or irritable bowel syndrome.
Pregnancy or breastfeeding
Prior gastrointestinal or bariatric surgery
Immunocompromised e.g., cancer treatment, bone marrow/organ transplant, immune deficiency, poorly controlled HIV/AIDS, prolonged use of steroids or other immunosuppressant medications
Antibiotic administration in the previous 30 days
Participation in other weight-loss programs in the previous 3 months.
Administration of pre/probiotic supplements or antibiotics.
Growth hormone administration in the previous 6 months
Must have access to a smartphone, tablet, computer, or other qualifying internet-enabled device and be able to follow instructions.
Individuals who are not proficient in English
Contraindications for MRI scanning, including Ferrous material implanted in or on the body, including flakes or filings, surgical clips, bullets, or electrical devices such as a pacemaker, or nonremovable ferrous jewelry (fillings in teeth and permanent retainers are permitted). Individuals with surgical pins or plates above the neck are excluded. Surgical pins or plates below the neck are exclusions, except when the material is fixed to bone, and considered acceptable by the Reference Manual for Magnetic Resonance Safety. Implants and Devices, 2020 Edition. Almost all recent orthopedic implants are made of materials that are not ferromagnetic and therefore are safe for scanning, and even though some screws are still made of ferromagnetic materials these are firmly screwed into bone. In cases where the material is unknown or deemed unsafe for scanning by the Reference Manual for Magnetic Resonance Safety. Implants and Devices, the participant will be excluded. History of eye injury involving metallic materials, shavings in eyes, or welding without a face mask. Lead/iron tattoos and tattoos performed by a nonprofessional artist if the pigment material is unknown. Claustrophobia (history of significant anxiety in closed places).
Back problem that would prevent the subject from laying still comfortably for up to 60 minutes.
Facility Information:
Facility Name
Robert Wood Johnson University Hospital
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
8220655
Citation
Holland AJ, Treasure J, Coskeran P, Dallow J, Milton N, Hillhouse E. Measurement of excessive appetite and metabolic changes in Prader-Willi syndrome. Int J Obes Relat Metab Disord. 1993 Sep;17(9):527-32.
Results Reference
background
PubMed Identifier
9526967
Citation
Fieldstone A, Zipf WB, Sarter MF, Berntson GG. Food intake in Prader-Willi syndrome and controls with obesity after administration of a benzodiazepine receptor agonist. Obes Res. 1998 Jan;6(1):29-33. doi: 10.1002/j.1550-8528.1998.tb00311.x.
Results Reference
background
PubMed Identifier
30569567
Citation
Proffitt J, Osann K, McManus B, Kimonis VE, Heinemann J, Butler MG, Stevenson DA, Gold JA. Contributing factors of mortality in Prader-Willi syndrome. Am J Med Genet A. 2019 Feb;179(2):196-205. doi: 10.1002/ajmg.a.60688. Epub 2018 Dec 19.
Results Reference
background
PubMed Identifier
26713776
Citation
Martinez Michel L, Haqq AM, Wismer WV. A review of chemosensory perceptions, food preferences and food-related behaviours in subjects with Prader-Willi Syndrome. Appetite. 2016 Apr 1;99:17-24. doi: 10.1016/j.appet.2015.12.021. Epub 2015 Dec 20.
Results Reference
background
PubMed Identifier
26425705
Citation
Zhang C, Yin A, Li H, Wang R, Wu G, Shen J, Zhang M, Wang L, Hou Y, Ouyang H, Zhang Y, Zheng Y, Wang J, Lv X, Wang Y, Zhang F, Zeng B, Li W, Yan F, Zhao Y, Pang X, Zhang X, Fu H, Chen F, Zhao N, Hamaker BR, Bridgewater LC, Weinkove D, Clement K, Dore J, Holmes E, Xiao H, Zhao G, Yang S, Bork P, Nicholson JK, Wei H, Tang H, Zhang X, Zhao L. Dietary Modulation of Gut Microbiota Contributes to Alleviation of Both Genetic and Simple Obesity in Children. EBioMedicine. 2015 Jul 10;2(8):968-84. doi: 10.1016/j.ebiom.2015.07.007. eCollection 2015 Aug.
Results Reference
background
PubMed Identifier
21722955
Citation
Purtell L, Sze L, Loughnan G, Smith E, Herzog H, Sainsbury A, Steinbeck K, Campbell LV, Viardot A. In adults with Prader-Willi syndrome, elevated ghrelin levels are more consistent with hyperphagia than high PYY and GLP-1 levels. Neuropeptides. 2011 Aug;45(4):301-7. doi: 10.1016/j.npep.2011.06.001. Epub 2011 Jul 1.
Results Reference
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PubMed Identifier
17636101
Citation
Dykens EM, Maxwell MA, Pantino E, Kossler R, Roof E. Assessment of hyperphagia in Prader-Willi syndrome. Obesity (Silver Spring). 2007 Jul;15(7):1816-26. doi: 10.1038/oby.2007.216.
Results Reference
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Understanding the Role of Gut Microbiota in Hyperphagia in Prader-Willi Syndrome
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